A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants with SHANK3 Haploinsufficiency

Status: Not yet recruiting

Phase of Trial: Phase I/II

Latest Information Update: 03 Nov 2024

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At a glance

Drugs JAG 201 (Primary)
Indications Telomeric 22q13 Monosomy Syndrome
Focus Adverse reactions; First in man
Sponsors Jaguar Gene Therapy

Most Recent Events

31 Oct 2024 New trial record

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